Trial in Australia, Europe will test heart drug in ALS patients
Study to see if trimetazidine improves function, life quality
A clinical trial in Australia and Europe will test whether trimetazidine, a medication commonly used to treat heart conditions, could help improve function and quality of life for amyotrophic lateral sclerosis (ALS) patients.
The trial, led by University of Queensland researcher Shyuan Ngo, PhD, and her team at the Australian Institute for Bioengineering and Nanotechnology, will start early next year. It will recruit about 150 patients across sites in Australia, the Netherlands, Belgium, Spain, and Italy.
Trimetazidine is prescribed in Europe for angina, a type of chest pain caused by reduced blood flow to the heart. Researchers say the medication may also support muscle and nerve health in ALS by helping cells optimize energy use.
“While ALS has traditionally been thought of as a disease of the brain or spinal cord, we now know that changes to a patient’s metabolism – or the rate they use energy – is linked to worse outcomes,” Ngo, who serves as co-director of the newly launched University of Queensland’s Centre for Motor Neuron Disease (MND) Research, said in a university news story. “If we can prove in this clinical trial that trimetazidine improves metabolism and function in ALS patients, doors will open for more research into how other existing drugs could be repurposed to treat ALS.”
Earlier trial tested safety
The study builds on an open-label Phase 2a MetFlex trial (NCT04788745), also led by Ngo’s team, showing that oral trimetazidine was safe and well tolerated in people with ALS. The trial enrolled 21 adults with ALS at sites in Australia and the Netherlands. Patients received nearly three months of trimetazidine treatment and were followed for another month.
Adverse events deemed possibly or probably related to trimetazidine included hot flushes, nausea, abnormal sensations, and fatigue, all of which disappeared after the patients stopped taking the medication. No one discontinued treatment due to side effects or died during the trial.
Efficacy data showed that the drug reduced markers of oxidative stress, a type of cell damage, and energy expenditure. However, the trial had a short duration and lacked a placebo group, which limited researchers’ ability to determine how trimetazidine influenced function and other clinical factors.
The new trial, funded by $2 million from FightMND and the Australian government, is part of a broader effort to explore how metabolic health affects ALS, and how existing medications might be repurposed to treat the disease.
“Modifying metabolic flexibility, or the balance between how a cell or tissue accesses sugars or fat as an energy source, is still an emerging area of research when it comes to MND and ALS, and the more we understand about it, the greater the chance we can find new treatments,” Ngo said.
The trial is one of three initiatives led by Ngo, who received $3.1 million from FightMND. Additional projects will investigate how ALS manifests differently in individuals and the role of muscle cell particles in ALS.
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